8 results
Supported by the observation from the Phase I studies in healthy volunteers, doses within a range of 50 to 500 mg are safe and well tolerated. This Phase II trial will be performed to:a. obtain proof of concept of BI 44370 TAb. perform dose finding…
Primary ObjectiveThe primary objective of this study is to evaluate glycogen clearance in muscle tissue samples collected pre and post alglucosidase alfa treatment in patients with late-onset Pompe disease.Secondary ObjectivesThe secondary…
The objective of the study is to gather more information about the long-term effect of enzyme therapy in patients with Pompe disease with a different severity of disease. The goal is also to set guideline for start and stop of treatment and to…
The overall objective of this study is to evaluate the efficacy and safety of neoGAA in treatment-naïve patients with LOPD as compared to alglucosidase alfa, when this is administered biweekly for a period of 49 weeks. Also, there is an open-label…
Primary: • To analyze the stability of the MHP using R.S.A to predict the long term survival.Secondary• To analyze the short term (1 year) safety of the MHP compared to the Stanmore hip replacement.• To analyze the short term (1 year) efficacy of…
This study intends to evaluate early clinical outcomes and survivorship of the G7 Acetabular System. Ease of instrument use will also be documented.The primary purpose of this study is to evaluate the clinical and radiographic performance of the G7…
The objective of the study is to asses the efficacy and safety of intravenous ATB200 co-administration with oral AT2221 by evaluating the changes in key clinical outcome measures (eg, motor, respiratory, fatigue) in adult subjects with late-onset…
Evaluate the effect of pediatric asthma eHealth care on the healthcare utilization, asthma outcomes and quality of life.